A Study of Panobinostat in Pediatric Patients With Solid Tumors Including MRT/ATRT

  • End date
    Dec 30, 2025
  • participants needed
  • sponsor
    Australian & New Zealand Children's Haematology/Oncology Group
Updated on 30 August 2021


This trial is evaluating the anti-tumor activity and side effects of panobinostat in treating patients with osteosarcoma, malignant rhabdoid tumor/atypical teratoid rhabdoid tumor (MRT/ATRT), and neuroblastoma.


This is an open label, phase II, multi-centre study evaluating the anti-tumor activity of continuous, low dose of panobinostat in patients with recurrent or refractory solid tumors stratified by primary histology into osteosarcoma, malignant rhabdoid tumor/atypical teratoid rhabdoid tumor (MRT/ATRT), and neuroblastoma.

Patients will be stratified at study entry by tumor type into three strata: osteosarcoma, MRT/ATRT and neuroblastoma [osteosarcoma and neuroblastoma arms are closed to enrolment]. Patients will be enrolled onto the study following completion of their conventional therapy including chemotherapy and/or radiation treatment and completion of a three-week wash out period.

Panobinostat will then be administered as a continuous oral dose (starting at a de-escalated dose of 8mg/m2 per day), for up to 12 courses, a total of 48 weeks. The minimum dose is 2mg/m2 per day. Dosing will follow a dose de-escalation or escalation scheme for each stratum which will be determined by biological effect of the drug (measured in patient peripheral blood samples) and levels of toxicity (measured by dose limiting toxicity and adverse events observed). Dose levels for subsequent enrolments in each strata will be based on the de-escalated or escalated dose in each cohort. The final dose per strata will be that which achieves significant biological effect with acceptable toxicity that is maintained for a 4 week period.

Patients or their parents/guardians will be required to maintain a drug diary to monitor drug usage throughout the trial. Patients will be followed for up to 2 years from completion of study therapy.

Condition Recurrent Brain Tumor, Childhood, Atypical Teratoid Rhabdoid Tumor, Atypical Teratoid/Rhabdoid Tumor, Rhabdoid Tumor
Treatment Panobinostat
Clinical Study IdentifierNCT04897880
SponsorAustralian & New Zealand Children's Haematology/Oncology Group
Last Modified on30 August 2021


Yes No Not Sure

Inclusion Criteria

Patients must be < 40 years of age
Patient must have been histologically diagnosed with osteosarcoma, neuroblastoma or MRT/ATRT at time of diagnosis or relapse. [osteosarcoma and neuroblastoma arms are closed to recruitment]
Patient disease is refractory to conventional therapy, in the case of osteosarcoma, neuroblastoma and MRT/ATRT or there is an absence of effective conventional therapy available in the case of ATRT. Patients must have stable disease (SD) or better following treatment with salvage therapy
Karnofsky performance level greater than or equal to 60% for patients 16 years of age and greater, OR Lansky performance levels greater than or equal to 60% for patients less than 16 years of age
Life expectancy of greater than 8 weeks
Fully recovered from acute toxic effects of all prior chemotherapy, immunotherapy or radiotherapy prior to entering study
Patients with CNS tumours who are receiving dexamethasone are on a stable/decreasing dose for at least 1 week
Adequate BM function
Adequate renal function
Adequate liver function
Adequate cardiac function
Adequate pulmonary function
Adequate CNS function - seizure free for at least 2 months
Adequate serum calcium, magnesium and potassium concentrations
If female and post-menarchal, pregnancy test must be negative
If of reproductive potential, have agreed to use effective contraceptive method
If female and lactating, have agreed not to breastfeed
Patient and/or their legal guardian have signed a written informed consent form

Exclusion Criteria

Have received myelosuppressive chemotherapy and/or biologic therapy within 3 weeks (4 weeks if prior nitrosourea)
Have received local palliative radiotherapy within 2 weeks
Have received craniospinal radiotherapy within 3 weeks
Have received greater than or equal to 50% radiation of the pelvis within 6 weeks
Have received other substantial BM radiation within 6 weeks
Have received growth factor(s) within 1 week
Are receiving enzyme inducing anticonvulsant therapy
Are receiving medications associated with prolongation of QTc interval
Are receiving hydrochlorothiazide
Are receiving metronidazole and/or disulfiram
Have uncontrolled sepsis
Have previously received panobinostat
Have symptoms of congestive heart failure, uncontrolled cardiac rhythm disturbance, or a QTc greater than or equal to 450msec
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